NDA Submitted for New Metastatic Urothelial Cancer Treatment

The NDA is based on data from the Phase 2 clinical trial which enrolled 96 patients with mUC and certain FGFR alterations.

Janssen announced the submission of a New Drug Application (NDA) for erdafitinib for the treatment of patients with locally advanced or metastatic urothelial cancer (mUC) and certain fibroblast growth factor receptor (FGFR) genetic alterations whose tumors have progressed after prior chemotherapy.

Erdafitinib is an investigational, once-daily oral pan-FGFR inhibitor. FRFRs are tyrosine kinases that when activated by alterations, can lead to increased tumor cell growth and survival.

The NDA submission was supported by data from the phase 2 BLC2001 clinical trial. The trial enrolled 96 patients with mUC and certain FGFR alterations. They were administered erdafitinib 8mg/day for five 28-day cycles with uptitration to 9mg/day if protocol-defined target serum phosphate level was not reached and if no significant treatment-related adverse events (TRAEs) occurred.

The primary endpoint was the percentage of patients with objective response rate (ORR), defined as complete or partial response based on RECIST v1.1 criteria.

The data showed a 42% confirmed ORR, of which 3% had complete response and 39% had partial response. There was a 70% complete ORR among those with prior immune checkpoint inhibitor treatment. Ten percent of patients discontinued due to TRAEs and no treatment-related deaths were reported.

“We look forward to working with the FDA in the agency’s review of the application as we believe erdafitinib will provide patients with an important therapeutic option,” said Peter Lebowitz, MD, PhD, Global Therapeutic Area Head, Oncology, at Janssen.